EU/3/08/559

Orphan designation

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in April 2014 on request of the sponsor.

On 5 September 2008, orphan designation (EU/3/08/559) was granted by the European Commission to Actelion Registration Limited, United Kingdom, for bosentan for the treatment of idiopathic pulmonary fibrosis.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Fibrosis is the formation of scar tissue that is part of the natural repair process of the body following tissue damage. Idiopathic pulmonary fibrosis consists of a chronic inflammation and progressive formation of fibrous tissue in the lungs. ‘Idiopathic’ means that the cause of the disease is unknown. The progressive formation of scars makes the lungs unable to work normally, reducing the transfer of oxygen from the air into the blood. Patients with idiopathic pulmonary fibrosis have a persistent cough, severe shortness of breath that gets worse and frequent lung infections.

Idiopathic pulmonary fibrosis is a long-term, debilitating and life-threatening disease.

At the time of designation, idiopathic pulmonary fibrosis affected approximately 1.9 in 10,000 people in the European Union (EU). This was equivalent to a total of around 95,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 502,800,000 (Eurostat 2008).

There is no treatment that stops the build-up of collagen. Available treatment is aimed at relieving symptoms and limiting damage. Several products with anti-inflammatory activity (products that limit the reaction of tissues against damage) were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation. Bosentan might be of potential significant benefit for the treatment of systemic sclerosis in particular with regard to efficacy in specific disease related complications. This benefit will have to be confirmed at the time of marketing authorisation and will be necessary to maintain the orphan status.

Bosentan opposes the effect of a substance called endothelin-1. This substance can cause narrowing of blood vessels and may also play an important role in the disease process of systemic sclerosis. Therefore, bosentan could play an important role in treating major complications of the disease.

At the time of submission of the application for orphan designation, clinical trials in patients with idiopathic pulmonary fibrosis were ongoing.

At the time of submission, bosentan was not authorised anywhere in the world for the treatment of idiopathic pulmonary fibrosis or designated as an orphan medicinal product elsewhere for this condition.

The product was authorised in the Community for the treatment of pulmonary arterial hypertension and the treatment of digital ulcers in patients with systemic sclerosis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 June 2008 recommending the granting of this designation.

the existence of alternative methods of diagnosis, prevention or treatment;

either the rarity of the condition (affecting not more than 5 in 10,000 people in the Community) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.